ION717 clinical trial completes patient enrolment
Autor: Eric Minikel (traducido por Joaquín Castilla) Ionis Pharmaceuticals ha anunciado (ver la carta original en inglés) esta mañana que ha completado la inscripción de PrProfile, su ensayo clínico de Fase 1/2a del ION717, un fármaco ASO diseñado para reducir los niveles de PrP en las enfermedades priónicas. El estudio ahora aparece como «Activo, sin […]
Scientists discover early brain alterations in people at risk of developing hereditary Creutzfeldt-Jakob disease
Author: Cristina Sampedro Prion diseases can occur spontaneously or genetically, the latter being caused by a mutation in the prion protein (PrP), which are rare but devastating neurodegenerative disorders that affect both humans and animals. The main types of genetic prion diseases are genetic Creutzfeldt-Jakob disease (gCJD), genetic Creutzfeldt-Jakob disease (gCJD), genetic Creutzfeldt-Jakob disease (gCJD), genetic [...]
Expansion and reactivation of Ionis PrProfile (ION717) clinical trial for people with symptomatic prion disease
Author: Joaquín Castilla Ionis Pharmaceuticals has just announced that the recruitment pause for its PrProfile (ION717) study for people with symptomatic prion disease has been lifted. Important information to note: Resumption of recruitment: After a planned pause, recruitment for the PrProfile clinical trial has resumed as of [...]
New international study looks for correlations between genetic variations and sporadic CJD
Author: Josu Galarza Scientists from around the world have collaborated on a genetic study that seeks to identify small changes in the genome that may be related to the clinical development and age of onset of sporadic Creutzfeldt-Jakob disease (sCJD). Prion diseases are classified as rare diseases due to their low incidence [...].
New strategies to understand and combat sporadic Creutzfeldt-Jakob disease through integration of genetic data
Autor: Nuno Anjo La enfermedad de Creutzfeldt-Jakob esporádica (sCJD) es una enfermedad neurodegenerativa rara, fatal y de rápida progresión, para la cual aún no existen tratamientos efectivos. Se caracteriza por el malplegamiento de la proteína priónica normal (PrPC) en una forma anómala llamada PrPSc o Prion, que se acumula en el cerebro, causando daño neuronal […]